Onasemnogene Abeparvovec in Type 1 Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis

Pascual‐Morena, Carlos; Cavero‐Redondo, Iván; Lucerón‐Lucas‐Torres, Maribel; Martínez‐García, Irene; Rodríguez‐Gutiérrez, Eva; Martínez‐Vizcaíno, Vicente

doi:10.1089/hum.2022.161

Cited by 17 publications

(13 citation statements)

References 48 publications

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“…Motor function decreased progressively in the cohorts of SMA’s natural history [ 33 , 34 ]. Conversely, the meta-analysis of data available in a 12-month follow-up estimated a proportion of CHOP-INTEND score ≥ 40 points of 87.28%, similar to the findings of other systematic reviews [ 16 , 17 ].…”

Section: Discussionsupporting

confidence: 83%

“…Previous systematic reviews have sought to assess the efficacy and safety of onasemnogene abeparvovec in relation to other technologies but have proved inconclusive with regard to some outcomes due to the impossibility of a connected network analysis and lack of data from all the studies available on the subject [ 13 , 14 ]. Other systematic reviews have focused on real-world studies, without statistical synthesis of the data [ 15 ], and on evaluating the efficacy—based only on the assessment of motor function—and safety of onasemnogene abeparvovec in patients with SMA [ 16 , 17 ]. Because it is an innovative and costly technology [ 18 ], an independent systematic review on the effectiveness and safety of onasemnogene abeparvovec in the main outcomes (survival, motor function and adverse events) is necessary to inform decision makers about the value of this technology considering the quality of clinical trials and the certainty of evidence.…”

Section: Introductionmentioning

confidence: 99%

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Efficacy and safety of onasemnogene abeparvovec for the treatment of patients with spinal muscular atrophy type 1: A systematic review with meta-analysis

Fernandes,

Krug,

Rodrigues

et al. 2024

PLoS ONE

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Background Onasemnogene abeparvovec has been approved for the treatment of spinal muscular atrophy 5q type 1 in several countries, which calls for an independent assessment of the evidence regarding efficacy and safety. Objective Conduct a meta-analysis to assess the efficacy and safety of onasemnogene abeparvovec in patients diagnosed with SMA type 1, based on the available evidence. Methods This article results from searches conducted on databases up to November 2022. Outcomes of interest were global survival and event-free survival, improvement in motor function and treatment-related adverse events. Risk of bias assessment and certainty of evidence were performed for each outcome. Proportional meta-analysis models were performed when applicable. Results Four reports of three open-label, non-comparative clinical trials covering 67 patients were included. Meta-analyses of data available in a 12-month follow-up estimate a global survival of 97.56% (95%CI: 92.55 to 99.86, I2 = 0%, n = 67), an event-free survival of 96.5% (95%CI: 90.76 to 99.54, I2 = 32%, n = 66) and a CHOP-INTEND score ≥ 40 points proportion of 87.28% (95%CI: 69.81 to 97.83, I2 = 69%, n = 67). Proportion of 52.64% (95%CI: 27.11 to 77.45, I2 = 78%, n = 67) of treatment-related adverse events was estimated. Conclusion The results indicate a potential change in the natural history of type 1 SMA, but the methodological limitations of the studies make the real extent of the technology’s long-term benefits uncertain.

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Section: Discussionsupporting

confidence: 83%

Section: Introductionmentioning

confidence: 99%

Efficacy and safety of onasemnogene abeparvovec for the treatment of patients with spinal muscular atrophy type 1: A systematic review with meta-analysis

Fernandes,

Krug,

Rodrigues

et al. 2024

PLoS ONE

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“… 17 , 18 The recently published real-world data mainly report short term data in cohorts with wider age and weight range than those used in clinical trials, also including infants previously treated with other disease modifying therapies. 10 , 11 , 12 , 19 , 20 , 21 , 22 , 23 , 24 , 25 …”

Section: Discussionmentioning

confidence: 99%

Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in naïve patients with spinal muscular atrophy and following switch from other therapies

et al. 2023

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“…37,38 Although tempting, these results cannot be compared with other gene therapies, such as onasemnogene abeparvovec. 39 This comparison is complicated by the necessarily limited sample size of some of the onasemnogene abeparvovec trials and the fact that participants were in different cohorts without randomization to one treatment or the other, which may lead to differences in baseline motor function, for example. However, as with onasemnogene abeparvovec, the preliminary results from NCT03779334 suggest that the greatest clinical benefit is achieved when risdiplam is administrated to presymptomatic participants.…”

Section: Discussionmentioning

confidence: 99%

Efficacy of risdiplam in spinal muscular atrophy: A systematic review and meta‐analysis

Pascual‐Morena,

Martínez‐Vizcaíno,

Cavero‐Redondo

et al. 2023

Pharmacotherapy

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ObjectivesThis systematic review and meta‐analysis aimed to assess the efficacy and safety of risdiplam on motor and respiratory function in spinal muscular atrophy (SMA).MethodsWe systematically searched Medline, Scopus, Web of Science, and the Cochrane Library from inception to March 2023. We included pre‐post studies that determined the effect of risdiplam on the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP‐INTEND), the 32‐item Motor Function Measure (MFM32), the Revised upper limb module (RULM), the Hammersmith Functional Motor Scale ‐ Expanded (HFMSE), respiratory function, and the proportion of risdiplam‐related adverse events in a population with SMA (phenotypes 1 and 2/3). Meta‐analyses were also performed where possible.ResultsEleven studies were included. After 12 months of treatment, 57% of participants with SMA1 achieved a CHOP‐INTEND score ≥40 points, and more than half were able to feed orally and had head control. In SMA2/3, MFM32, RULM, and HFMSE increased by 2.09 (1.17, 3.01), 1.73 (1.25, 2.20), and 1.00 (0.40, 1.59) points, respectively. Efficacy on respiratory function in SMA2/3 was inconsistent. Finally, 16% of participants experienced adverse events, but serious adverse events could not be quantified due to a lack of cases.ConclusionThe limited available evidence suggests that risdiplam is an effective and safe drug for the treatment of SMA. In addition, long‐term clinical benefit may be partly determined by the stage of disease at which treatment is initiated.

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Onasemnogene Abeparvovec in Type 1 Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis

Cited by 17 publications

References 48 publications

Efficacy and safety of onasemnogene abeparvovec for the treatment of patients with spinal muscular atrophy type 1: A systematic review with meta-analysis

Efficacy and safety of onasemnogene abeparvovec for the treatment of patients with spinal muscular atrophy type 1: A systematic review with meta-analysis

Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in naïve patients with spinal muscular atrophy and following switch from other therapies

Efficacy of risdiplam in spinal muscular atrophy: A systematic review and meta‐analysis

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